Results 141 to 150 of about 3,524,002 (263)

Gene therapy for lysosomal storage diseases: Current clinical trial prospects. [PDF]

Front Genet, 2023
Kido J, Sugawara K, Nakamura K.
europepmc   +1 more source

Mutant KRAS‐m6A Epitranscriptome Axis Promotes Colorectal Cancer and is a Therapeutic Target

Advanced Science, EarlyView.
Mutant KRAS‐m6A axis promotes colorectal cancer (CRC) progression. Mutant KRAS stabilizes METTL3, leading to increased m6A‐modified BCL9L mRNA and translation of BCL9L protein. BCL9L in turn mediates TGF‐β secretion to induce differentiation of Treg and an immunosuppressive microenvironment.
Danyu Chen, Pingmei Huang, Ruizhi Tao, Fenfen Ji, Qinyao Wei, Lok Hin Ko, Lianxin Zhou, Huarong Chen, Alvin Cheung, Wei Kang, Ko Fai To, Jun Yu, Chi Chun Wong   +12 more
wiley   +1 more source

Metabolomic Insights into Lysosomal Storage Diseases: An Untargeted View. [PDF]

Metabolites
Di Carlo G, Tommolini ML, Frisco A, Spalluto G, Foley D, Zucchelli M, Cufaro MC, Cicalini I, Bucci I, Federici L, De Laurenzi V, Pieragostino D, Rossi C.   +12 more
europepmc   +1 more source

SIRT7‐Mediated H2BK120 Succinylation Drives Aberrant Mitophagy in Sepsis‐Associated Cognitive Dysfunction

Advanced Science, EarlyView.
Sepsis triggers hippocampal SIRT7 loss, promoting histone H2B succinylation and activation of the PD‐1/PD‐L1–PINK1 axis. This metabolic‐epigenetic cascade drives aberrant mitophagy and neuronal injury, ultimately causing cognitive deficits. Targeting SIRT7‐dependent succinylation offers a potential strategy to protect brain function during sepsis ...
Na Meng, Jiateng Zhou, Xiaoyu Guo, Ting Hong, Xuelian Li, Xingyu Wei, Chanhua Zhang, Xixue Zhang, Songbin Liu, Yulin Zhang, Liangfang Yao, Lina Huang, Weidong Gu   +12 more
wiley   +1 more source

Nonimmune Hydrops Fetalis and Lysosomal Storage Diseases

Pediatrics and Neonatology, 2013
Carlo Bellini
doaj   +1 more source

Application of Mesenchymal Stromal Cells and Their Exosomes in Neurodegenerative Diseases and Lysosomal Storage Diseases. [PDF]

Cells
Ayupova AI, Sidorova AS, Luzina EA, Sufianov AA, Sufianova GZ, Zaynutdinov AM, Rizvanov AA, Solovyeva VV.   +7 more
europepmc   +1 more source

Multifunctional Nanovaccine‐Radiotherapy Synergy Drives Anti‐Tumor Immunity via Multi‐Cascade Cell Death for Pancreatic Cancer Therapy

Advanced Science, EarlyView.
Machine‐learning‐driven transcriptomic analysis identifies FSTL3 as a key tumor‐microenvironment regulator in desmoplastic pancreatic adenocarcinoma. A cRGD‐modified liposome co‐loading gemcitabine and siFSTL3 serves as an irradiation‐triggered in situ nanovaccine. It synergizes with radiotherapy to induce ferroptosis and immunogenic cell death through
Chengyi Huang, Yushu Han, Xiang Liu, Chuanhao Wang, Xiaofei Zhu, Di Chen, Yangyang Gong, Liang Chen, Wenjuan Chen, Zhenyuan Miao, Bufu Tang, Huojun Zhang   +11 more
wiley   +1 more source

Population Pharmacokinetic/Pharmacodynamic Modeling of Therapeutic Enzymes in Lysosomal Storage Diseases. [PDF]

Clin Pharmacokinet
Barzel I, van der Ploeg AT, Pijnappel WWMP, van der Kuy PHM, van den Hout JMP, Preijers T.   +5 more
europepmc   +1 more source

Self‐Propelled HPB@Lip@AB Nanomotors Ameliorate Dry Eye Disease

Advanced Science, EarlyView.
A self‐propelled HPB@Lip@AB nanomotor system is developed for dry eye disease (DED) therapy, facilitating rapid penetration across ocular surface barriers while integrating hydrogen therapy with multi‐enzyme‐like antioxidant activity. The nanoplatform restores mitochondrial function, suppresses oxidative stress, inflammation, and apoptosis, and ...
Jing Li, Xi Wan, Xinru Su, Zechuan Li, Yue Wang, Ying Wang, Ruofei Li, Huangzhao Wei, Jianbin Zhang, Shuai Wang   +9 more
wiley   +1 more source

The Sphingolipid Balance and Endothelial Dysfunction in Lysosomal Storage Diseases: Shared Mechanisms in Gaucher, Niemann-Pick and Fabry Disease. [PDF]

Int J Mol Sci
Nekrasova A, Kutsev S, Shestopalov A.
europepmc   +1 more source